Carrier of Duchenne Muscular DystrophyNCT01712152
Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study
An observational study of Carrier of Duchenne Muscular Dystrophy, run by Hospital Rudolfstiftung at 1 site in Austria.
Trial summary
Carriers of Duchenne muscular dystrophy (DMD) often have no severe symptoms of the scelet muscles, but they may develop a poor heart function due to the involvement of the heart muscle. Ultrasound of the heart is recommended, but it can detect a cardiac affection only after the heart has already become weaker. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Carrier of Duchenne Muscular Dystrophy
- How many people
- 20 participants
- Where
- 1 siteAustria
- Study dates
- Oct 2012to Sep 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Women only
- No healthy volunteers
- Adult, Older adult
Study population: data base of carriers of Duchenne muscular dystrophy
Who may be able to join
- genetic and/or histological identification as a carrier of DMD age above 18 years able and willing to conform to the requirements of the study provided written informed consent exclusion of pregnancy in women of childbearing potential
Who cannot join
- Claustrophobia Excessive obesity to an extent where CMR cannot be performed Chronic renal failure with a GFR <30 ml/min/1,73m² Implanted pacemakers/defibrillators Severe arrhythmia Inability to cooperate during the CMR Known intolerance to gadolinium Positive pregnancy test Unable or unwilling to conform to the study protocol
Trial locations and contacts
1 site in 1 country.
About this study
Carriers of Duchenne muscular dystrophy (DMD) often have no severe symptoms of the scelet muscles, but they may develop a poor heart function due to the involvement of the heart muscle. Ultrasound of the heart is recommended, but it can detect a cardiac affection only after the heart has already become weaker. Cardiac magnetic resonance tomography can detect myocardial fibrosis already at preserved heart function and may facilitate early therapy. In our study, diagnosed carriers of DMD will undergo examinations of the heart by blood tests, EKG, heart ultrasound and magnetic resonance at enrollment and after one year in order to assess the extent of cardiac affection and its association with the clinical development.
- Conditions
- Carrier of Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, carrier, cardiac MRI, myocardial fibrosis, female
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
T1 mapping
T1 mapping by MOLLI sequences will be performed and compared between study entry and one year follow-up
Measured: 1 year
Secondary outcomes (1)
left ventricular function
left ventricular function on MRI will be compared between study entry and one year follow-up
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2012 (estimated)
- Study startOct 2012
- Primary completionApr 2014
- Study completionSep 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Carrier of Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital Rudolfstiftung
- Sponsor type
- University, hospital or other
- Collaborators
- Oesterreichische Muskelforschung
- Responsible party
- Principal investigator, Paul Wexberg,MD, Hospital Rudolfstiftung
- Investigators
- Paul Wexberg, MD, Study director, Hospital Rudolfstiftung
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01712152
- Sponsor's protocol ID
- EK 11-228- 0112
- Status last verified
- Oct 2016
- First submitted
- 19 Oct 2012
- Last updated
- 4 Oct 2016
- Results posted
- No
Publications
Wexberg P, Avanzini M, Mascherbauer J, Pfaffenberger S, Freudenthaler B, Bittner R, Bernert G, Weidinger F. Myocardial late gadolinium enhancement is associated with clinical presentation in Duchenne muscular dystrophy carriers. J Cardiovasc Magn Reson. 2016 Sep 22;18(1):61. doi: 10.1186/s12968-016-0281-y.
PubMed 27660108
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study
- ClinicalTrials.gov ID
- NCT01712152
- Acronym / sponsor ID
- EK 11-228- 0112
- Conditions
- Carrier of Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, carrier, cardiac MRI, myocardial fibrosis, female
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Hospital RudolfstiftungUniversity, hospital or other
- Collaborators
- Oesterreichische Muskelforschung
- Study start
- Oct 2012
- Primary completion
- Apr 2014 (actual)
- Study completion
- Sep 2015 (actual)
- First posted
- 23 Oct 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Carriers of Duchenne muscular dystrophy (DMD) often have no severe symptoms of the scelet muscles, but they may develop a poor heart function due to the involvement of the heart muscle. Ultrasound of the heart is recommended, but it can detect a cardiac affection only after the heart has already become weaker. Cardiac magnetic resonance tomography can detect myocardial fibrosis already at preserved heart function and may facilitate early therapy. In our study, diagnosed carriers of DMD will undergo examinations of the heart by blood tests, EKG, heart ultrasound and magnetic resonance at enrollment and after one year in order to assess the extent of cardiac affection and its association with the clinical development.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- data base of carriers of Duchenne muscular dystrophy
Key inclusion criteria (1 listed)
- genetic and/or histological identification as a carrier of DMD age above 18 years able and willing to conform to the requirements of the study provided written informed consent exclusion of pregnancy in women of childbearing potential
Key exclusion criteria (1 listed)
- Claustrophobia Excessive obesity to an extent where CMR cannot be performed Chronic renal failure with a GFR <30 ml/min/1,73m² Implanted pacemakers/defibrillators Severe arrhythmia Inability to cooperate during the CMR Known intolerance to gadolinium Positive pregnancy test Unable or unwilling to conform to the study protocol
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
T1 mapping
Time frame: 1 year
T1 mapping by MOLLI sequences will be performed and compared between study entry and one year follow-up
1 secondary outcome
left ventricular function
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2016.
- Derived from this study: PubMed 27660108Wexberg P, Avanzini M, Mascherbauer J, Pfaffenberger S, Freudenthaler B, Bittner R, Bernert G, Weidinger F. Myocardial late gadolinium enhancement is associated with clinical presentation in Duchenne muscular dystrophy carriers. J Cardiovasc Magn Reson. 2016 Sep 22;18(1):61. doi: 10.1186/s12968-016-0281-y.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paul Wexberg, MD study director
Hospital Rudolfstiftung
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Carrier of Duchenne Muscular Dystrophy, run by Hospital Rudolfstiftung at 1 site in Austria.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
T1 mapping (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01712152, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Oct 2016.